Gene Therapy for ACM Due to a PKP2 Pathogenic Variant

A Phase 1/2 Study of the Safety and Efficacy of LX2020 Gene Therapy in Patients With Arrhythmogenic Cardiomyopathy Due to a Plakophilin-2 Pathogenic Variant

This is a Phase 1/2, first-in-human, open-label, intravenous, dose-escalating, multicenter trial that is designed to assess the safety and tolerability of LX2020 in adult patients with PKP2-ACM

Selected Inclusion Criteria:

• Adults with a clinical diagnosis of ACM meeting the 2010 revised Task Force Criteria (TFC)
• Genetic testing documenting a pathogenic or likely pathogenic variant in PKP2
• Frequent premature ventricular complexes (PVCs)
• Implantable cardioverter-defibrillator (ICD) implantation ≥ 12 weeks prior to the pre-screening MRI
• Left ventricular ejection fraction ≥ 40%

Selected Exclusion Criteria:

• Evidence of variant(s) in addition to PKP2 that meets the standard criteria to be considered pathogenic or likely pathogenic for ACM
• Other cardiac abnormalities as specified in the protocol
• New York Heart Association Functional Class IV at the time of consent
• History of prior gene transfer therapy